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The REPLACE Registry for Cholbam® (Cholic Acid)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03115086
Recruitment Status : Recruiting
First Posted : April 14, 2017
Last Update Posted : August 8, 2022
Sponsor:
Information provided by (Responsible Party):
Travere Therapeutics, Inc.

Study Description
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Brief Summary:
This is a prospective, observational, non-interventional patient registry study designed to document product safety and effectiveness outcomes for 10 years in patients treated with Cholbam, including those who have been using Cholbam for at least 30 days (existing users) and those who are first-time initiators of Cholbam.

Condition or disease Intervention/treatment
Bile Acid Synthesis Disorders Drug: Cholbam

Detailed Description:
No experimental intervention is involved. Patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.
Study Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 55 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 10 Years
Official Title: A Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated With Cholbam® (Cholic Acid)
Actual Study Start Date : July 10, 2017
Estimated Primary Completion Date : July 2038
Estimated Study Completion Date : July 2039

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Existing User
Patients who have been using Cholbam for at least 30 days
 Drug: Cholbam
Cholbam prescribed according to the approved label.
New User
First-time initiators of Cholbam
 Drug: Cholbam
Cholbam prescribed according to the approved label.


Outcome Measures
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Primary Outcome Measures :
  1. Number of participants with worsening cholestasis [ Time Frame: 10 Years ]
    Worsening cholestasis will be identified by measuring direct total bilirubin concentration in blood and will be defined as a 25% increase from previous measurement if the total bilirubin is >1mg/dL.

  2. Number of participants with new-onset cholestasis [ Time Frame: 10 Years ]
    Patients with new-onset cholestasis will be identified by detecting abnormal direct bilirubin concentration >1mg/dL or direct bilirubin greater than 20% of the total bilirubin if total bilirubin is >5mg/dL.

  3. Number of participants with steatorrhea leading to poor growth [ Time Frame: 10 Years ]
    Steatorrhea leading to poor growth in children, which will be defined as a decrease in growth percentiles from the original percentile at enrollment to the registry study.

  4. Changes in serum levels of fat-soluble vitamins [ Time Frame: 10 Years ]
    Fat-soluble vitamin (A, D, E, K) deficiencies will be identified by comparing results from serum assays (Vitamin A: Retinol, Vitamin D: 250HD2 + 250HD3, Vitamin K: Serum Vitamin K, Vitamin E: Serum Vitamin E) for each vitamin with standard ranges.

  5. Neuropathic signs or symptoms related to fat-soluble vitamin deficiencies [ Time Frame: 10 Years ]
    Neuropathic signs or symptoms related to fat-soluble vitamin deficiencies (peripheral neuropathy, cerebellar ataxia) will be assessed by the Investigator.

  6. Number of participants with growth failure [ Time Frame: 10 Years ]
    Growth failure that the physician judges to be attributable to malabsorption.

  7. Death [ Time Frame: 10 Years ]
    AEs and SAEs leading to death will be recorded. The relatedness of death to Cholbam or disease progression will be recorded.

  8. Adverse effects on pregnancy, pregnancy outcomes, and infant status [ Time Frame: 10 Years ]
    Adverse effects on pregnancy, pregnancy outcomes, and infant status will be recorded.

  9. All Adverse Events (AEs) and Serious AEs (SAEs) [ Time Frame: 10 years ]
    All other AEs and SAEs will be collected.


Secondary Outcome Measures :
  1. Changes in Cholbam dosing regimens and reasons for any dose modifications or treatment discontinuations [ Time Frame: 10 years ]
  2. Changes from baseline in weight [ Time Frame: 10 years ]
    For patients aged 18 years of or less, these data will be converted into Z-scores based on the WHO growth standards for infants aged 0-2 years and the Centers for Disease Control and Prevention growth standards for children 2-20 years. Growth failure will be defined as a decrease in percentiles from the original percentile at entry point into the registry study.

  3. Changes from baseline in length/height [ Time Frame: 10 years ]
    For patients aged 18 years of or less, these data will be converted into Z-scores based on the WHO growth standards for infants aged 0-2 years and the Centers for Disease Control and Prevention growth standards for children 2-20 years. Some patients will be adult or will have achieved final height. Growth failure will be defined as a decrease in percentiles from the original percentile at entry point into the registry study.

  4. Changes from baseline in head circumference in infants [ Time Frame: 10 years ]
    For patients aged 18 years of or less, these data will be converted into Z-scores based on the WHO growth standards for infants aged 0-2 years and the Centers for Disease Control and Prevention growth standards for children 2-20 years. Growth failure will be defined as a decrease in percentiles from the original percentile at entry point into the registry study.

  5. Age-appropriate developmental milestones in infants [ Time Frame: 10 years ]
    Measured by WHO Motor Development Milestones

  6. Changes in prothrombin time (PT) [ Time Frame: 10 years ]
  7. Changes in international normalized ratio (INR) [ Time Frame: 10 years ]
  8. Changes in albumin [ Time Frame: 10 years ]
  9. Changes in bilirubin [ Time Frame: 10 years ]
  10. Changes in direct bilirubin [ Time Frame: 10 years ]
  11. Changes in alanine aminotransferase (ALT) [ Time Frame: 10 years ]
  12. Changes in aspartate aminotransferase (AST) [ Time Frame: 10 years ]
  13. Changes in gamma-glutamyl transferase (GGT) [ Time Frame: 10 years ]
  14. Changes in alkaline phosphatase [ Time Frame: 10 years ]
  15. Presence or absence of urinary bile acids and levels of bile acid intermediaries and urinary bile alcohol [ Time Frame: 10 years ]
  16. All indications for which Cholbam has been prescribed [ Time Frame: 10 years ]
    Description of all indications for which Cholbam has been prescribed


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
All patients with a diagnosis for which Cholbam is indicated are eligible for inclusion in the Registry.
Criteria

Inclusion Criteria:

  1. Male and female patients, of any age.
  2. The patient and/or the patient's parent/legal guardian is willing and able to provide signed informed consent, and the patient, if less than 18 years of age, is willing to provide assent as appropriate and in accordance with local regulatory, IRB, and EC requirements.
  3. The patient has a diagnosis for which Cholbam is indicated.
  4. The patient is or will be treated with Cholbam at the time of signing the informed consent form (ICF) (enrollment).

Exclusion Criteria:

1. Patients who, by judgement of the Investigator, will not be able to comply with the requirements of the protocol will be excluded

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03115086


Contacts
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Contact: Travere Medical Information 1-877-659-5518 medinfo@travere.com

Locations
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Sponsors and Collaborators
Travere Therapeutics, Inc.
Investigators
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Study Director: Feriandas Greblikas, MD Senior Medical Director
More Information
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Additional Information:

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Responsible Party: Travere Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03115086    
Other Study ID Numbers: 031CHO15001
First Posted: April 14, 2017    Key Record Dates
Last Update Posted: August 8, 2022
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Travere Therapeutics, Inc.:
Bile Acid Synthesis Disorder
Zellweger Spectrum Disorder
Peroxisomal Disorder
 Cholic Acid
Cholbam
The REPLACE Registry